Now play­ing catch-up to blue­bird, Ver­tex and CRISPR send in their pitch for blood dis­or­der cell ther­a­py

If all things go ac­cord­ing to plan, the FDA could start re­view­ing the first-ever fil­ing for a CRISPR ther­a­py this No­vem­ber.

Ver­tex and CRISPR Ther­a­peu­tics say they have wrapped up their lengthy talks with the agency and got­ten reg­u­la­tors on board for a rolling re­view be­gin­ning lat­er this year — with the sub­mis­sion pack­age due to be com­plete in the first quar­ter of 2023.

While the part­ners once hoped that they would be the first to bring a once-and-done ther­a­py to the US mar­ket for sick­le cell dis­ease (SCD) and trans­fu­sion-de­pen­dent be­ta tha­lassemia (TDT), blue­bird bio beat them to the punch with the re­cent FDA ap­proval of Zyn­te­glo. The fil­ing al­so marks a slight de­lay to ear­li­er plans of fil­ing in late 2022, al­though it wasn’t en­tire­ly un­ex­pect­ed.

As then-SVB Se­cu­ri­ties an­a­lyst Rick Bi­enkows­ki not­ed in June, the last pa­tient dosed in the study that formed the ba­sis of Ver­tex and CRISPR’s pitch “will not have reached the 12-month fol­low-up time­point un­til Jan­u­ary 2023.” If the FDA re­quires that every pa­tient make it over that line, he wrote, then BLA fil­ings would need to be pushed to ear­ly 2023 — which end­ed up be­ing the case.

Al­though the com­pa­nies didn’t spec­i­fy the rea­son, they did pre­vi­ous­ly men­tion that the FDA was look­ing for an en­tire year of fol­low-up.

The part­ners added, how­ev­er, that they’re on track to file for ap­proval in the EU and UK by the end of 2022.

CTX001, now dubbed ex­agam­glo­gene au­totem­cel or exa-cel, was born out of a part­ner­ship be­tween Ver­tex and CRISPR that be­gan in 2015. In a ma­jor sign of con­fi­dence, Ver­tex paid CRISPR $900 mil­lion cash two years ago to take an ad­di­tion­al 10% of fu­ture sales from the ther­a­py, which is an au­tol­o­gous cell ther­a­py fea­tur­ing ex vi­vo gene ed­its done with CRISPR/Cas9 tech­nol­o­gy.

Specif­i­cal­ly, a pa­tient’s own hematopoi­et­ic stem cells are edit­ed to pro­duce high lev­els of fe­tal he­mo­glo­bin. The con­cept is sim­i­lar to blue­bird’s Zyn­te­glo, ex­cept that Zyn­te­glo con­tains the he­mo­glo­bin sub­unit be­ta gene and the ed­its are de­liv­ered with a lentivi­ral vec­tor.

At their last up­date, Ver­tex and CRISPR re­port­ed that of 44 pa­tients with TDT who re­ceived exa-cel, 42 had stopped red blood cell trans­fu­sions. As for the sick­le cell dis­ease tri­al, all 31 pa­tients have had no se­vere va­so-oc­clu­sive crises.

It re­mains to be seen whether safe­ty con­cerns re­lat­ed to the con­di­tion­ing agent pa­tients need to re­ceive be­fore get­ting exa-cel would cause prob­lems in the re­view.

Forge Bi­o­log­ics’ cGMP Com­pli­ant and Com­mer­cial­ly Vi­able Be­spoke Affin­i­ty Chro­matog­ra­phy Plat­form

Forge Biologics has developed a bespoke affinity chromatography platform approach that factors in unique vector combinations to streamline development timelines and assist our clients in efficiently entering the clinic. By leveraging our experience with natural and novel serotypes and transgene conformations, we are able to accelerate affinity chromatography development by nearly 3-fold. Many downstream purification models are serotype-dependent, demanding unique and time-consuming development strategies for each AAV gene therapy product1. With the increasing demand to propel AAV gene therapies to market, platform purification methods that support commercial-scale manufacturing of high-quality vectors with excellent safety and efficacy profiles are essential.

Feng Zhang (Susan Walsh/AP Images)

In search of new way to de­liv­er gene ed­i­tors, CRISPR pi­o­neer turns to mol­e­c­u­lar sy­ringes

Bug bacteria are ruthless.

Some soil bacteria have evolved tiny, but deadly injection systems that attach to insect cells, perforate them and release toxins inside — killing a bug in just a few days’ time. Scientists, on the other hand, want to leverage that system to deliver medicines.

In a paper published Wednesday in Nature, MIT CRISPR researcher Feng Zhang and his lab describe how they engineered these syringes made by bacteria to deliver potential therapies like toxins that kill cancer cells and gene editors. With the help of an AI program, they developed syringes that can load proteins of their choice and selectively target human cells.

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Cedric Ververken, Confo Therapeutics CEO

Dai­ichi Sankyo inks $183M dis­cov­ery deal with GPCR biotech for CNS tar­get

Belgian biotech Confo Therapeutics has landed $183 million, plus potential royalties, in a drug-discovery deal with Daiichi Sankyo.

Early Thursday, Confo Therapeutics put out word of the deal that will be focused on small molecule antagonists to go after an undisclosed target that the company says is associated with CNS diseases.

Confo CEO Cedric Ververken told Endpoints News that Daiichi originally reached out to learn about the biotech’s technology. He added that Confo, founded in 2015, will use its platform to drug a GPCR target that Daiichi has struggled with internally.

Dif­fu­sion to hand Nas­daq spot to EIP Phar­ma for PhI­Ib de­men­tia study of ex-Ver­tex drug

One of the more than a dozen bidders for Diffusion Pharmaceuticals’ spot on Nasdaq has prevailed.

Boston biotech EIP Pharma will merge with Diffusion in an all-stock deal, with plans to start a Phase IIb clinical trial in the coming months in a common form of dementia with no approved treatments. The combined company will be renamed CervoMed.

The nine-year-old privately-held EIP is working on a former Vertex drug that it will test in a 160-person Phase IIb in patients with dementia with Lewy bodies, or DLB. The National Institute on Aging is expected to fund that trial with a $21 million grant. With the reverse merger, slated for closing in the middle of this year, EIP will be funded through that readout in the second half of 2024. EIP’s equity and debt holders will own about 77.25% of the combined company.

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Luke Miels, GSK chief commercial officer

GSK picks up Scynex­is' FDA-ap­proved an­ti­fun­gal drug for $90M up­front

GSK is dishing out $90 million cash to add an antifungal drug to its commercial portfolio, in a deal spotlighting the pharma giant’s growing focus on infectious diseases.

The upfront will lock in an exclusive license to Scynexis’ Brexafemme, which was approved in 2021 to treat a yeast infection known as vulvovaginal candidiasis, except in China and certain other countries where Scynexis already out-licensed the drug.

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Mathai Mammen, FogPharma's next CEO

Math­ai Mam­men hands in J&J's R&D keys to lead Greg Ver­dine’s Fog­Phar­ma 

In the early 1990s, Mathai Mammen was a teaching assistant in Greg Verdine’s Science B46 course at Harvard. In June, the former R&D head at Johnson & Johnson will succeed Verdine as CEO, president and chair of FogPharma, the same month the seven-year-old biotech kickstarts its first clinical trial.

After leading R&D at one of the largest drugmakers in the world, taking the company through more than half a dozen drug approvals in the past few years, not to mention a Covid-19 vaccine race, Mammen departed J&J last month and will take the helm of a Cambridge, MA biotech attempting to go after what Verdine calls the “true emperor of all oncogenes” — beta-catenin.

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FDA ap­proves Nar­can opi­oid over­dose re­ver­sal spray for over-the-counter sale

The FDA today approved Emergent BioSolutions’ Narcan brand naloxone nasal spray for over-the-counter sales. The nod was expected and comes on the heels of a unanimous 19-0 advisory committee vote in favor of approval last month.

The move to OTC means the opioid overdose reversal agent will now be available on grocery, convenience and gas stations shelves, as well as potentially for purchase online.

CSL CEO Paul McKenzie (L) and CMO Bill Mezzanotte

Q&A: New­ly-mint­ed CSL chief ex­ec­u­tive Paul McKen­zie and chief med­ical of­fi­cer Bill Mez­zan­otte

Paul McKenzie took over as CEO of Australian pharma giant CSL this month, following in the footsteps of long-time CSL vet Paul Perreault.

With an eye on mRNA, and quickly commercializing its new, $3.5 million-per-shot gene therapy for hemophilia B, McKenzie and chief medical officer Bill Mezzanotte answered some questions from Endpoints News this afternoon about where McKenzie is going to take the company and what advances may be coming to market from CSL’s pipeline. Below is a lightly edited transcript.

Boehringer re­ports ro­bust sales led by type 2 di­a­betes and pul­monary drugs, promis­es more to come high­light­ing obe­si­ty

Boehringer Ingelheim reported human pharma sales of €18.5 billion on Wednesday, led by type 2 diabetes and heart failure drug Jardiance and pulmonary fibrosis med Ofev. Jardiance sales reached €5.8 billion, growing 39% year over year, while Ofev took in €3.2 billion, notching its own 20.6% annual jump.

However, Boehringer is also looking ahead with its pipeline, estimating “In the next seven years the company expects about 20 regulatory approvals in human pharma.”